Gruppo San Donato’s Post

🏆 The European Research Council (ERC) has announced the winners of the 2024 Consolidator Grants, including three San Raffaele researchers: 👩🔬 Daniela Cesana, for IRCCS Ospedale San Raffaele, with her project “TalesfromDeath” on the effectiveness of gene therapy in patients with genetic disorders and tumors 👨🔬 Alessio Cantore, for UniSR, with his project “HEPAGENE” on the genetic engineering of hepatocytes to treat diseases of liver metabolism in children 👩🔬 Daniela Latorre, for #UniSR, with her project “AUTO-T-NERVES” on inflammatory neuropathies characterized by an abnormal activation of the immune system In the field of Life Sciences, 4 projects have been funded in Italy, 3 of which were awarded to San Raffaele. The ERC Consolidator Grant provides support to excellent Principal Investigators at the career stage at which they may still be consolidating their own independent research team or programme, with up to EUR 2 million for 5 years. With these last 3, San Raffaele reaches 34 ERC grants awarded since 2007, when the Programme was first launched. Read the full news at https://lnkd.in/dygV_Vra San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) #ERCCoG #unisr #universitàvitasalute #sanraffaele #innovatingthroughknowledge #universitàsanraffaele #unisrmi

This year’s #Nobel Prize in #Physiology or #Medicine #2024 celebrates the groundbreaking work of Victor Ambros and Gary Ruvkun for their discovery of #microRNA and its crucial role in post-transcriptional #gene regulation. Their discovery unveiled a fundamental mechanism that has been shaping gene regulation for hundreds of millions of years, enabling the evolution of increasingly complex organisms. #MicroRNA plays a critical role in ensuring the normal development of #cells and #tissues. Without it, #genetic research shows that these processes would not occur as they should. The implications of this discovery are profound. Abnormal regulation of microRNAs can lead to #cancer, and #mutations in microRNA-coding genes in humans have been linked to conditions such as congenital hearing loss, as well as eye and skeletal disorders. This remarkable achievement not only advances our understanding of gene regulation but also opens new avenues for #medical #research and therapies. 🔗 The link for detailed information about the research: https://lnkd.in/gyQyPQQq #NobelPrize #PhysiologyOrMedicine #GeneRegulation #microRNA #ScientificDiscovery #MedicalBreakthrough #GeneticResearch #NobelLaureates

Celebrating the 2024 Nobel Prize for Groundbreaking Research on miRNAs! 🏆🔬 The 2024 Nobel Prize in Physiology or Medicine was awarded to Victor Ambros and Gary Ruvkun for their discovery of microRNAs (miRNAs), which play a crucial role in regulating gene expression. Ambros first identified miRNA in C. elegans in 1993, while Ruvkun later discovered another miRNA and demonstrated its evolutionary importance. The 2024 Nobel Prize was awarded for pioneering research on microRNAs (miRNAs), small yet powerful molecules that regulate gene expression. This discovery has revolutionized our understanding of cellular processes and has opened new avenues for treating diseases like cancer, neurodegenerative disorders, and viral infections. By unveiling the critical role miRNAs play in gene regulation, this work is pushing the boundaries of medical science and personalized medicine! #NobelPrize2024 #miRNA #GeneTherapy #MedicalBreakthrough #Biotech

Our Gene Therapy Research Team have made an exciting step forward towards developing a new way to target the lungs more effectively, with the aim of improving treatment for cystic fibrosis. During a recent lab tour, we had the honour of hosting Team Simon Foundation for Cystic Fibrosis and Cure4 Cystic Fibrosis who have generously supported this research. The team explained the complex process of designing new vectors and were excited to share they had narrowed the search down from an initial 64 million vector options, to a small subset that appears to successfully target the lungs. Children's Medical Research Institute received the major Holckner Family CF Impact Grant from Cure4 Cystic Fibrosis Foundation in 2023. It is the second major grant to our researchers, thanks to fundraising efforts of the Team Simon Foundation who have a long history with CMRI. Read more on the recent visit here: https://lnkd.in/g3GkvZcF

wow Great innovation & discover Nobel Prize : The seminal discovery of microRNAs – recognised by the 2024 Nobel Prize in Physiology or Medicine – was unexpected and unveiled a new dimension of gene regulation. Gene regulation by microRNA, first revealed by this year’s medicine laureates Victor Ambros and Gary Ruvkun, has been at work for hundreds of millions of years. This mechanism has enabled the evolution of increasingly complex organisms. We know from genetic research that cells and tissues do not develop normally without microRNAs. Abnormal regulation by microRNA can contribute to cancer, and mutations in genes coding for microRNAs have been found in humans, causing conditions such as congenital hearing loss, eye and skeletal disorders. Mutations in one of the proteins required for microRNA production result in the DICER1 syndrome, a rare but severe syndrome linked to cancer in various organs and tissues. The 2024 Nobel Prize in Physiology or Medicine has been awarded to Victor Ambros and Gary Ruvkun for the discovery of microRNA and its role in post-transcriptional gene regulation.

𝐄𝐧𝐠𝐢𝐧𝐞𝐞𝐫𝐞𝐝 𝐏𝐨𝐥𝐲𝐦𝐞𝐫𝐬 𝐒𝐡𝐨𝐰 𝐏𝐫𝐨𝐦𝐢𝐬𝐞 𝐟𝐨𝐫 𝐒𝐲𝐬𝐭𝐞𝐦𝐢𝐜 𝐦𝐑𝐍𝐀 𝐃𝐞𝐥𝐢𝐯𝐞𝐫𝐲 𝐭𝐨 𝐋𝐮𝐧𝐠𝐬: 𝐏𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥 𝐈𝐦𝐩𝐥𝐢𝐜𝐚𝐭𝐢𝐨𝐧𝐬 𝐟𝐨𝐫 𝐆𝐞𝐧𝐞𝐭𝐢𝐜 𝐋𝐮𝐧𝐠 𝐃𝐢𝐬𝐞𝐚𝐬𝐞𝐬 Scientists engineered nanoparticles capable of delivering genetic material to lung cells with high efficiency when administered intravenously. Based on biodegradable polymers, this delivery method has demonstrated promising performance in both human lung cells and mouse models, potentially overcoming the limitations of current gene therapy approaches for lung diseases. Read more in our interview with Erin Kavanagh, Johns Hopkins Medicine Thanks to Christos Evangelou, PhD, BCMAS for the writeup! https://lnkd.in/eQJeGC2D #crisprmedicinenews #crisprmedicine #genedelivery #genetherapy #cysticfibrosis #lungdisease #mRNA

Researchers at the University of Buffalo have successfully reversed the brain abnormalities in infant mice with FOXG1 syndrome 🧠 using gene therapy. In the detailed study published in Molecular Therapy - Methods & Clinical Development, a group of scientists used adeno-associated viral vector 9️⃣ to deliver a functional version of FOXG1 gene into the brains of infant mice 💉 . ✨ This approached has successfully led to restoration of the corpus callosum, a critical structure connecting the brain's hemispheres that is underdeveloped in FOXG1 syndrome. Additionally, the therapy also increased the size of the dentate gyrus in the hippocampus, normalized the number of oligodendrocytes precursor cells, and improved myelination. These results highlight the therapeutic potential of this approach for treating FOXG1 syndrome in infants and opened doors to treatment for other neurodevelopmental disorders with similar neural abnormalities. #braindisorder #raredisease #developmental #neurodegenerative #FOXG1 #myelination #genetherapy #AAV #viralvector #UniversityofBuffalo #UBuffalo https://lnkd.in/d7WwXA9m

Congratulations to the winners of the 2024 Nobel Prize in Physiology or Medicine, honored for their groundbreaking discovery of microRNA and its role in post-tanscriptional gene regulation. In the early 1990s, Ambros and Ruvkun uncovered a new class of tiny RNA molecules—microRNAs—that play a crucial role in regulating gene expression. Their work revealed how microRNAs control the timing and activity of specific genes, providing a critical mechanism for cellular differentiation and function in multicellular organisms. The research, which began in the humble roundworm C. elegans, actually unlocked a new dimension in biology, reshaping our understanding of gene regulation. Today, we know that over a thousand microRNAs exist in the human genome, influencing everything from cell specialization to disease processes, with applications spanning developmental biology, cancer research and beyond! #NobelPrize2024 #MedicalResearch #MicroRNA #GeneRegulation #RodonBiologics

All individuals with two copies of the APOE4 gene develop signs of Alzheimer's Researchers have found that over 95% of individuals over 65 years old who have two copies of the APOE4 gene -APOE4 homozygotes- show biological characteristics of Alzheimer's pathology in the brain or biomarkers of this disease in cerebrospinal fluid and PET scans. The study, published in Nature Medicine, also concludes that those individuals homozygous for APOE4 also develop the disease earlier than those with other variants of the APOE gene. These findings suggest that having two copies of the APOE4 gene could represent a new genetic form of Alzheimer's disease, as explained by the author. #ScienceMission #ScienceNewsHighlights https://lnkd.in/g5DrNimk